No National Pay Policy on Anemia Drugs Likely

WASHINGTON -- The Centers for Medicare and Medicaid Services (CMS) announced it likely won't change its payment policy on erythropoiesis stimulating agents (ESAs) used to treat anemia in kidney patients.

Currently CMS payment policies for the drugs including epoetin alfa (Procrit and Epogen) and darbepoetin alfa (Aranesp) vary by region.

In a draft decision released Wednesday, CMS indicated it will not issue a national coverage determination (NCD) for ESAs because of a lack of evidence on whether their use makes kidney transplant patients less likely to reject the organ.

"Despite an exhaustive search, we identified no high-quality, randomized clinical trials that were of sufficient design, duration, and power to definitely determine that ESAs provided clinical benefits other than increasing hemoglobin..." CMS wrote in the draft decision.

The agency will seek comments on its proposal to not issue a coverage decision, and will issue a final decision in June.

Wednesday's decision is just what a Medicare Evidence Development & Coverage Advisory Committee (MedCAC) decided in January when it voted that there isn't enough evidence to make any scientific decision on ESAs.

"This is just a reminder how many data there aren't," MedCAC chairman Clifford Goodman, PhD, said after that meeting.

Anemia is a serious problem for kidney disease patients and one that gets worse as the disease progresses. The destruction of renal function in chronic kidney disease patients causes a loss of erythropoietin, which can cause anemia.

ESAs work by replicating erythropoietin, a protein made by the kidney that stimulates bone marrow cells to produce red blood cells. That increase in the number of red blood cells often makes therapeutic transfusions -- the traditional treatment for anemic kidney disease patients -- unnecessary.

Some say that repeated blood transfusions lead to the development of antibodies that fight a transplanted kidney, so eliminating transfusions will make a patient's body more likely to accept the new kidney. And one way to eliminate transfusions is by using ESAs.

But in its proposed memo, CMS officials said they found no evidence to definitively support that claim.

"The evidence for transfusion reduction is limited because of the absence of validated criteria for transfusion, the absence of defined study protocols for transfusion, and the use of noninferiority (or equivalence) study designs that lacked a placebo arm," CMS officials wrote.

The FDA approved the first ESA in 1989.

In recent years, the safety of ESAs has come under scrutiny, and a number of studies were published that linked ESAs to an increased risk of MI, heart failure, stroke, blood clots, and even death.

In March 2007, the FDA required ESAs to carry a black box warning, recommending they be used at the lowest dose necessary to prevent the need for transfusions.

The FDA convened an advisory panel to revisit safety issues of darbepoetin alpha and in October 2010, an FDA panel voted overwhelmingly to recommend that the agency allow continued use of the drug for chronic kidney disease patients who are not on dialysis.

CMS' ultimate decision will have ramifications for those on private insurance as well as Medicare, since private health insurers tend to take their coverage cues from Medicare.

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